Pediatric Clinical Trials Are Often Abandoned, Unpublished

Choosing to enroll your child in a clinical trial is a personal and ethically complex decision. Maybe your child has a genetic condition or rare type of cancer that has no available treatment. Or maybe you’re a parent living in an area of the world where certain infectious diseases are endemic, and enrolling your child in a vaccine trial could lessen the chances of your child developing a serious, life-threatening infection.

In recent years, federal legislation has encouraged more pediatric trials to help establish drug dosing, safety and efficacy since most therapies, treatments and medical devices prescribed for children have not been tested in children. But a new study suggests a significant number of pediatric clinical trials are either stopped before they’re completed, or if completed, their results go unpublished. The findings appear in the journal Pediatrics.

“No matter what the focus of the trial, there is some inherent risk for any child to be involved in a trial,” said Dr. Natalie Pica, a resident at Boston Children’s Hospital and co-author on the study, in an interview. “We owe it to those participants to conduct those trials ethically and disseminate that information.”

Previous research has shown similar problems among clinical trials in adults, but these practices have been less studied in pediatric populations.

The most commonly cited reason for a trial ending early was difficulty with patient recruitment. Interestingly, funding issues were the least likely to be cited as reasons for trial discontinuation. But when funding was cited as a factor, trials funded by academia were more likely to be discontinued compared to those funded by industry.

Of the 455 completed trials, 136–or 30%–had not published findings in a scientific journal nearly five years after the trial ended. Pica and Bourgeois calculated that those unpublished trials included 69,165 pediatric participants.

The FDA Amendments Act of 2007 mandated registration and reporting of clinical trial results of medicines and devices subject to FDA regulation. But researchers can fulfill that requirement by posting results on ClinicalTrials.gov rather than publishing in scientific journals, according to the authors. Still, only 42 of the 136 clinical trials that went unpublished in journals posted their data on ClinicalTrials.gov.

The Pediatrics study found that even though trials funded by industry were more likely to be completed, their results were more than twice as likely to go unpublished two or three years after the end of a clinical trial compared with trials sponsored by academia.

Beyond ethical and scientific considerations, the discontinuation of pediatric clinical trials reveals another concern for academia, industry and nonprofit scientific organizations alike: money. Clinical trials are expensive, and discontinued trials represent a huge a huge area of inefficiency and waste of financial and human resources.

“There has to be some sort of oversight, whether they’re funded by industry or academia, and have feasible goals and available resources to achieve those proposed goals,” Pica said.

That type of oversight is taking shape in Europe, where new rules rolled out by the European Medicines Agency in January 2015 aim to make public more clinical trial data than ever before.

I am a freelance science writer who seeks to explore the intersection of health and humanity. I have written about public and global health, neuroscience, genomics, regenerative medicine, aging, rare diseases, cancer and science policy, among other topics. I hold an MA in S...